Clinical trial patients to Congress: 'Stop acting like babies'

The visitors’ entrances to the National Institutes of Health are blocked off with traffic cones. The usually teeming campus, spread over rolling green hills in the Washington, D.C. suburb of Bethesda, Md., is quiet. The patients inside aren’t so quiet, however.

Justin Smith and his daughter McKenna are both furious at Congress. “They need to stop acting like babies,” says McKenna, who turns 13 on Friday.

McKenna slipped into a clinical trial under the wire this week, arriving on the NIH campus just as the government shutdown closed down the start of medical studies like the one she is taking part in. The Cape Coral, Fla., girl is lucky — trials that are already underway will continue. But there’s a hold on starting new ones until Congress can come to an agreement on funding the federal government’s operations.

Michelle Langbehn of Auburn, Calif., wasn’t so fortunate. Langbehn, 30 and the mother of a toddler, has a rare form of cancer and had hoped to get into an NIH-sponsored clinical trial of a so-called orphan drug — one approved for use against rare diseases — called Cometriq. That was called off this week.

“Every day counts, every second counts,” says Langbehn, who’s been through surgery, radiation and several rounds of chemotherapy.

Justin Smith and his daughter McKenna, 12, take a break at the Children's Inn on the campus of the National Institutes of Health. They are furious that the government shutdown has affected McKenna's care at NIH.Maggie Fox / NBC News

"I've always promised my daughter that I refuse to let her grow up without a mom and, I'm going to make that happen no matter what I have to do.”

"There's going to be an impact on health and there's going to be people who might die, and that's not fair,” says Dr. Louis Weiner, director of the Lombardi Comprehensive Cancer Center at Georgetown University.

NIH has furloughed 13,698 of its 18,646 employees — 73 percent in all. That includes staff taking care of patients like McKenna, who has a rare genetic condition called neurofibromatosis Type 1. It causes tumors to grow all over the insides of her body.

She’s in a trial to see if a cancer drug called AZD6244 can stop the tumors that are wrapping themselves around her nerves, collapsing her lung, squeezing her esophagus and causing her constant pain. Without it, the only option is surgery to remove tumors that will just return again and again, and she’s been told she probably won’t live past 18 without a new, good treatment.

“This research is very, very vital to my daughter and to thousands of others who come here,” Justin Smith says. “We are both angry.”

The NIH says no new studies will be started during a government shutdown. Six new trials were due to start next week — now they won’t. “Approximately 200 new patients per week will be deferred admission to the NIH Clinical Center during the shutdown,” NIH says. Of these, 30 are children like McKenna.

“We’ve waited a year to come here,” Smith added, “And now that we are here, it’s like a ghost town.”

That not only means there's a skeleton staff, but also that there are no cafeterias open, nowhere to get a bite to eat between hour after hour of MRIs, blood tests and treatments. The Smiths are lucky to be staying in the Children’s Inn at NIH, a non-profit center run entirely on private donations, so it’s not at the mercy of government funding.

There, McKenna can play with a therapy dog and her dad can cook meals in their kitchen. He’s worried that if the government shutdown goes on for too long, even ongoing trials like the one McKenna is in will close.

“We have to be back up here in a couple of weeks, then back again in a couple of months,” Smith says. “We are afraid they are going to say ‘You can’t come back.’ This is our last resort.”

Republican members of the House of Representatives say they won’t pass legislation to keep funding the federal government unless they get concessions from Democrats on the Affordable Care Act. Democrats in the Senate and President Barack Obama say no way.

“To be stopped at the edge over ideology and BS!” Smith says. “I’d love to have my senator come up and shake my daughter’s hand and apologize to her for what they are doing.”

NIH isn't the only federal health agency affected. The Centers for Disease Control and Prevention, the Food and Drug Administration and food stamp programs are, too. Medicare and Medicaid won't be, because they are funded differently.

NIH is currently running nearly 500 different clinical trials, about half of them for cancer treatments. Patients enrolled at studies at other hospitals around the country are probably not affected by the shutdown, especially if the trials are paid for by drug companies or universities. But NIH often studies the people with the rarest diseases and the least hope — conditions that don’t justify a drug company’s investment.

And NIH pays for studies being run by 300,000 researchers around the country and the world.

NIH had already been stinging from something else Congress did — the sequester. This was the measure that cut 5 percent from the federal budget across the board. The NIH slashed $1.5 billion from its budget. “This means every area of medical research will be affected,” NIH said in a statement.